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Gene editing technology shrinks tumors in mice, showing promise for cancer treatment

Wes Venteicher
| Monday, May 1, 2017, 11:00 p.m.

University of Pittsburgh cancer researchers shrunk tumors and extended the lives of mice using genome-editing technology that could make cancer treatment more effective and less unpleasant.

Researchers used CRISPR-Cas9, which enables editing of the genetic code in DNA, to target “breakpoints” where genes have fused with one another in a process associated with the initiation of many cancers, said Dr. Jian-Hua Luo, director of Pitt's High Throughput Genome Center and the lead author of a study on the technique. The journal Nature Biotechnology published the study Monday.

The breakpoints provide junctures in the genetic code where researchers can introduce a virus to kill cancer cells while leaving healthy cells unharmed, Luo said.

His research team tested the technique in mice with transplanted human prostate and liver cancer cells, according to the study. Tumors shrunk up to 30 percent in mice who had received the treatment, and the treated mice lived through the eight-week study period without their cancer metastasizing. In mice who didn't receive the treatment, tumors grew and metastasized; all died before the study ended.

While conventional chemotherapy treatments target the proteins involved in cancer's growth, often causing collateral damage, the CRISPR-based method aims to shut off cancer's development earlier in the process, Luo said.

“We could treat cancer in a new way that would take out the cancer control center, effectively,” he said.

The technique could be applied to other forms of cancer that stem from gene fusion, he said. He said about 90 percent of cancers are associated with some type of fusion, although many are associated with multiple fusions and other gene mutations.

While the technique in the study only shrunk tumors, Luo said he thinks it has the potential to eliminate them. He said he hopes to begin testing in humans within five years.

Wes Venteicher is a Tribune-Review staff writer. Reach him at 412-380-5676, wventeicher@tribweb.com or via Twitter @wesventeicher.

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